Trial boosts hopes for new cholesterol busting drug

A drug that boosts the body's natural ability to lower cholesterol could help protect against heart attacks and strokes, according to new research.

The drug was able to cut levels of "bad" cholesterol in the bloodstream by 40% within two weeks, doctors discovered during an early-stage clinical trial of moderately overweight volunteers.

Because the medication works differently to statins, the most common cholesterol-lowering drugs, it may be most effective for those patients who are unable to tolerate statins because of their side effects.

Researchers at the University of California, San Francisco, and the Karolinska Institute in Stockholm designed the drug to mimic a thyroid hormone that accelerates the body's ability to flush unhealthy cholesterol, known as low density lipoprotein, from the body.

In the trial 24 volunteers aged between 18 and 60, with a body mass index of 25 to 35 and high cholesterol, were divided into three groups. One group received no medication but had blood cholesterol levels monitored for the duration of the trial, while the others received different doses of the drug, called KB2115. After two weeks blood tests revealed that the drug lowered cholesterol and increased the speed it was processed by the liver.

The trial follows a series of animal studies which found the drug removes cholesterol from arteries and transports it to the liver where it is converted into bile. The animal studies showed the drug also countered diabetes and obesity by reducing cholesterol.

Attempts to develop thyroid hormone mimicking drugs have been held up because they produced harmful side effects, such as a raised heart rate. Based on their first study in humans, the researchers believe their drug avoids this by working almost exclusively in the liver.

Writing in the journal Proceedings of the National Academy of Sciences, the scientists claim none of the patients experienced harmful side effects, but larger scale trials are needed to confirm this. One possibility that needs to be ruled out is the danger of causing hyperthyroidism, which can cause dangerous weight loss, depression and heart disorders.

More than 3 million people take statins in England, but according to the British Heart Foundation more than 100,000 still die of heart disease in Britain each year. Some patients are unable to take statins because of their side effects, which include diarrhoea, headaches and rashes.

"We need other types of drugs to attack this problem. Using thyroid mimics is an entirely different approach, and I think one with great promise for treating high cholesterol and probably other conditions such as obesity and diabetes," said Professor John Baxter, a co-author on the paper at the Diabetes Centre at University of California, San Francisco.